Charlotte and Gwenyth Gray, seen here in 2015 with their parents Gordon and Kristen Gray, have a rare disorder known as Batten disease. While a new drug approved Thursday won’t help the strain of the disease the girls have, their mother — a Naperville native — is optimistic it could lead to other breakthroughs. (Photo from Larissa Block / Handout)
A former Naperville resident says a new drug on the market raises hope for parents of children who suffer from a rare and always fatal degenerative condition known as Batten disease.
Once healthy toddlers, children with Batten disease quickly decline, losing the ability to walk, talk or feed themselves. Life expectancy is only 8 to 12 years.
On Thursday, the U.S. Food and Drug Administration approved BioMarin Pharmaceutical Inc.’s enzyme replacement therapy – Brineura – to treat the slow loss of walking ability in pediatric patients 3 years and older with the form of Batten known as late infantile neuronal ceroid lipofuscinosis type 2, or CLN2
Batten disease came to the attention of the Naperville community when three local moms – Casey McCormick, Bianca Morin and Whitney Robbins – raised $400,000 to fight a rare disease afflicting the daughters of a childhood friend.
In a matter of months, the group pulled together a fundraiser to benefit the foundation created by former Naperville resident Kristen Kaiser Gray and her husband, Hollywood film producer Gordon Gray, on behalf of their daughters, Charlotte and Gwenyth Gray.
Although Brineura cannot help Charlotte and Gwenyth (they have different form known as CLN6), Kaiser Gray said the news is "extremely exciting" for the Batten community.
"Families have been waiting for something," she said. "It just opens doors to the possibilities."
Kaiser Gray said Dr. Emily de los Reyes, attending pediatric neurologist at Nationwide Children’s Hospital in Ohio and principal investigator for the Brineura studies, also worked established the protocols for the gene therapy program that Charlotte and Gwenyth have been receiving for the past year.
Thursday was both happy and sad for Downers Grove residents Tracy and Jennifer VanHoutan, parents of two children affected by CLN2 – Liane, 11, whose condition is too far along for Brineura to benefit, and Noah, who passed away last year at age 11.
Their Hope for Noah foundation created in 2009 helped fund the research that led to the clinical trials needed to bring the drug for FDA approval.
"It’s been a roller coaster of a day. We are thrilled it was approved," Tracy VanHoutan said. "At the same time, it’s a little bittersweet. I wished it could have helped my children."
CLN2 disease is a rare inherited disorder that primarily affects the nervous system. Signs and symptoms, which typically begin between ages 2 and 4, include language delay, recurrent seizures and difficulty coordinating movements such as sitting and walking.
The condition often requires the use of a wheelchair by late childhood and most do not survive past their teens.
"The FDA is committed to approving new and innovative therapies for patients with rare diseases, particularly where there are no approved treatment options," said Dr. Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research in a statement. "Approving the first drug for the treatment of this form of Batten disease is an important advance for patients suffering with this condition."